Breakthrough for cystic fibrosis

Published: August 11, 2006 | 4588th good news item since 2003

Scientists said this week they have new insight into how cells break down the faulty proteins produced by cystic fibrosis.

The discovery that the cells break down the misshapen proteins before the proteins can mend themselves is important information that might form the basis for new CF treatments.

Douglas Cyr and his research team at the University of North Carolina Cystic Fibrosis Pulmonary Research and Treatment Center noted that cystic fibrosis (CF), a genetic disease, causes the body to produce a misshapen form of the crucial protein cystic fibrosis transmembrane conductance regulator (CFTR). CFTR regulates the fluidity of the mucous in epithelial cells of the respiratory and intestinal tracts.

This mucous is too thick in people with CF, and the respiratory and digestive problems that result prove fatal in early adulthood.

Cyr said earlier work revealed that badly folded CFTR could correct itself in cell culture if it was treated with compounds known as chemical chaperones or simply left alone so it could fully open up. Unfortunately, bad CFTR gets sent to the molecular trash bin before this can happen.

The team discovered that the cell disposes of unwanted molecules by marking them with a protein called ubiquitin that tells an organelle called a proteasome to pick it up and shred it. Cyr said that if this process could be interrupted, it could extend the lifetime of misshapen CFTR and give it enough time to assume a functional shape.